When Business, Science Collide

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When Business, Science Collide

DENVER – When medical research and business mix it may raise eyebrows – if not serious ethical questions – but scientists say the two need each other to ensure that medical advances are delivered to the public.

"I think it's important to appreciate that there's an important role at both ends," said Dr. Harry Malech, head of genetic immunotherapy at the National Institute of Allergy and Infectious Disease in Bethesda, Maryland. "There are very few things that have gone directly from basic science to being widely available to people in the community to treat their disease process. Industry is essential for translating these things."

Malech was among researchers representing academia, government, and industry at the American Academy of Gene Therapy conference who discussed their respective positions on Saturday for choosing non-profit versus for-profit settings to do their research, and why neither can be painted as a villain or an angel.

Clinical trials involving gene therapy have received an unprecedented amount of public and government attention since the September death of an Arizona teenager who was participating in a gene therapy experiment at the University of Pennsylvania.

Food and Drug Administration officials found many shortcomings in the protocol of the Penn trial.

Conflict of interest was a main concern because Dr. James Wilson, director of the Penn lab, held an equity stake in Genovo, a gene therapy company that partially funded the experiment during which Jesse Gelsinger, the Arizona teenager, died.

Industry representatives defended Wilson, saying they believe the incident was a symptom of inexperience, and not willful negligence.

The university announced in late May that the program, which has been under scrutiny by the FDA and was the subject of Senate hearings, will be limited in the future to experiments on animals. The FDA had already halted all human gene therapy experiments at the lab, and the university declared that Wilson will have only a peripheral role in gene therapy experiments that will take place in other departments of the university.

The Penn case was an example of a role reversal, because academic researchers are not as experienced with clinical trials as those in industry; therefore, a significant learning curve exists, said Stephen Chang, chief scientific officer of San Diego, California-based gene-therapy firm Canji.

While pharmaceutical companies have evolved alongside the FDA as it refined regulations, academic researchers are new to the world of clinical trial procedures.

"Sitting here in this room are some of the experts in the field, and we'll have to educate the other way – so we become (teachers) of the professors," Chang said.

"They don't teach you how to run a clinical trial in medical school," said Douglas Jolly, vice president of scientific affairs at Chiron in San Diego.

Historically, academic and government researchers have focused on more fringe, rare diseases in which larger pharmaceutical houses – ever searching for indications that will reach a large market – have no interest.

For example, Dr. Alain Fischer of Necker University Hospital in Paris, successfully used gene therapy to treat four boys with a rare disease called severe combined immunodeficiencies, an immune system disease that requires patients to live in sterile "bubbles." Without the treatment, the boys would have died within one year. Three of the boys have been healthy for more than a year.

"That is a project that no large pharmaceutical and some biotechs (would undertake)," Chang said. "I would have never attempted to breach that subject with my management. But it was of tremendous value to the field."

Often, advances in rare diseases can translate into useful methods for more widespread ailments.

Malech – who studies a rare ailment called chronic granulomatous disease that affects about 1,000 people in the United States – said since industry is concerned with larger markets, some diseases stand the threat of becoming "orphaned."

"CGD is a very good example of an orphan disease," Malech said. "There are no companies currently interested in developing treatments for CGD – it's not going to happen and that's true of a whole variety of relatively rare diseases. It's an example of why (this) research is important, because it's from there that many things get developed."

Connie Eaves, professor of medical genetics and deputy director of the Terry Fox Laboratory at the British Columbia Cancer Agency in Vancouver, agreed that industry and academia work in harmony to bring medical products to the public. The important role of academia, she said, lies in its freedom from the pressures of turning a profit.

"Observation A and observation B ultimately come together in the context of rigorous scientific method coupled with creativity, not driven by (profit), in a way that in industry would not," Eaves said. "Also, the youngest minds come to academia – I hate to admit it, but that's really where science is born."